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NAMSR News: Stem Cell Breakthrough // Patient-specific Therapies for Type 1 Diabetes
A team of scientists from the New York Stem Cell Foundation Research Institute and Columbia University Medical Center, also in New York, claim to have created the first disease-specific embryonic stem cell line with two sets of chromosomes.
The research began in 2006 as part of an effort to make patient-specific embryonic stem cell lines from people who have type 1 diabetes.
The process behind this innovation is called somatic cell nuclear transfer (SCNT). It involves taking unfertilized donor oocytes - the immature egg cells used in reproduction - and adding to them the nuclei of adult skin cells taken from the patient.
Stem cell experiments for this project initially took place at Harvard University in Cambridge, MA, with skin biopsies from the patients being performed at Columbia University Medical Center.
However, isolating cell nuclei from skin biopsies was not possible in the federally funded Columbia labs. As a solution, the New York Stem Cell Foundation Research Institute (NYSCF) opened its own privately-funded laboratory to complete the research - now the largest independent stem cell laboratory in the US.
By 2008, all of the research had been moved to the NYSCF lab, as Massachusetts restrictions prevented Harvard from obtaining oocytes.
In an associated news release, the NYSCF say that "the reprogramming of skin cells from a type 1 diabetes patient by SCNT has long been sought," but that progress within this field has been stymied by difficulties in obtaining oocytes for research, as well as an incomplete understanding of the biology of human oocytes.
LAB-CREATED INSULIN-PRODUCING BETA CELLS COULD OFFER A CURE FOR TYPE 1 DIABETES
People with type 1 diabetes are deficient in insulin and have high blood sugar levels because they lack insulin-producing beta cells. Therefore, if beta cells can be successfully produced from stem cells, they could be transplanted into diabetic patients as a treatment and potential cure. As the stem cells are made from the patient's own skin cells, they would all match the patient's DNA.
In 2011, the scientists reported that they had created the first embryonic stem cell line from human skin using nuclear transfer. However, the stem cells produced had three sets of chromosomes and could not be used as a treatment.
But the scientists have now been able to create a patient-specific embryonic stem cell line that has two sets of chromosomes, which is the normal number in human cells.
Dr. Dieter Egli, the NYSCF scientist who led the research, says: "From the start, the goal of this work has been to make patient-specific stem cells from an adult human subject with type 1 diabetes that can give rise to the cells lost in the disease. By reprogramming cells to a pluripotent state and making beta cells, we are now one step closer to being able to treat diabetic patients with their own insulin-producing cells."
However, this is not the end of this research; the success in producing the patient-specific cells only marks the completion of the first phase of the project. The next step is to develop strategies that prevent the immune systems of type 1 diabetes patients from attacking their own beta cells.
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